Consistent improvement with eculizumab across muscle groups in myasthenia gravis

Objective: To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti-acetylcho...

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Autores: Mantegazza, Renato, O'Brien, Fanny L., Yountz, Marcus, Howard, James F. Jr., REGAIN Study Group, Casasnovas Pons, Carlos
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2020
País:España
Institución:Universidad de Barcelona
Repositorio:Dipòsit Digital de la UB
OAI Identifier:oai:diposit.ub.edu:2445/196128
Acceso en línea:https://hdl.handle.net/2445/196128
Access Level:acceso abierto
Palabra clave:Anticossos monoclonals
Malalties neuromusculars
Malalties autoimmunitàries
Monoclonal antibodies
Neuromuscular diseases
Autoimmune diseases
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spelling Consistent improvement with eculizumab across muscle groups in myasthenia gravisMantegazza, RenatoO'Brien, Fanny L.Yountz, MarcusHoward, James F. Jr.REGAIN Study GroupCasasnovas Pons, CarlosAnticossos monoclonalsMalalties neuromuscularsMalalties autoimmunitàriesMonoclonal antibodiesNeuromuscular diseasesAutoimmune diseasesObjective: To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups. Methods: Patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis were randomized 1:1 to receive either placebo or eculizumab during the REGAIN study (NCT01997229). Patients who completed REGAIN were eligible to continue into the open-label extension trial (NCT02301624) for up to 4 years. The four domain scores of each of the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale recorded throughout REGAIN and through 130 weeks of the open-label extension were analyzed. Results: Of the 125 patients who participated in REGAIN, 117 enrolled in the open-label extension; 61 had received placebo and 56 had received eculizumab during REGAIN. Patients experienced rapid improvements in total scores and all four domain scores of both the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale with eculizumab treatment. These improvements were sustained through 130 weeks of the open-label extension. Interpretation: Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.American Neurological Association2020info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersionapplication/pdfhttps://hdl.handle.net/2445/196128Articles publicats en revistes (Ciències Clíniques)reponame:Dipòsit Digital de la UBinstname:Universidad de BarcelonaInglésReproducció del document publicat a: https://doi.org/10.1002/acn3.51121Annals of Clinical and Translational Neurology, 2020, vol. 7, num. 8, p. 1327-1339https://doi.org/10.1002/acn3.51121cc-by-nc-nd (c) Mantegazza, Renato et al., 2020https://creativecommons.org/licenses/by-nc-nd/4.0/info:eu-repo/semantics/openAccessoai:diposit.ub.edu:2445/1961282026-05-27T06:46:51Z
dc.title.none.fl_str_mv Consistent improvement with eculizumab across muscle groups in myasthenia gravis
title Consistent improvement with eculizumab across muscle groups in myasthenia gravis
spellingShingle Consistent improvement with eculizumab across muscle groups in myasthenia gravis
Mantegazza, Renato
Anticossos monoclonals
Malalties neuromusculars
Malalties autoimmunitàries
Monoclonal antibodies
Neuromuscular diseases
Autoimmune diseases
title_short Consistent improvement with eculizumab across muscle groups in myasthenia gravis
title_full Consistent improvement with eculizumab across muscle groups in myasthenia gravis
title_fullStr Consistent improvement with eculizumab across muscle groups in myasthenia gravis
title_full_unstemmed Consistent improvement with eculizumab across muscle groups in myasthenia gravis
title_sort Consistent improvement with eculizumab across muscle groups in myasthenia gravis
dc.creator.none.fl_str_mv Mantegazza, Renato
O'Brien, Fanny L.
Yountz, Marcus
Howard, James F. Jr.
REGAIN Study Group
Casasnovas Pons, Carlos
author Mantegazza, Renato
author_facet Mantegazza, Renato
O'Brien, Fanny L.
Yountz, Marcus
Howard, James F. Jr.
REGAIN Study Group
Casasnovas Pons, Carlos
author_role author
author2 O'Brien, Fanny L.
Yountz, Marcus
Howard, James F. Jr.
REGAIN Study Group
Casasnovas Pons, Carlos
author2_role author
author
author
author
author
dc.subject.none.fl_str_mv Anticossos monoclonals
Malalties neuromusculars
Malalties autoimmunitàries
Monoclonal antibodies
Neuromuscular diseases
Autoimmune diseases
topic Anticossos monoclonals
Malalties neuromusculars
Malalties autoimmunitàries
Monoclonal antibodies
Neuromuscular diseases
Autoimmune diseases
description Objective: To assess whether eculizumab, a terminal complement inhibitor, improves patient- and physician-reported outcomes (evaluated using the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale, respectively) in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis across four domains, representing ocular, bulbar, respiratory, and limb/gross motor muscle groups. Methods: Patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis were randomized 1:1 to receive either placebo or eculizumab during the REGAIN study (NCT01997229). Patients who completed REGAIN were eligible to continue into the open-label extension trial (NCT02301624) for up to 4 years. The four domain scores of each of the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale recorded throughout REGAIN and through 130 weeks of the open-label extension were analyzed. Results: Of the 125 patients who participated in REGAIN, 117 enrolled in the open-label extension; 61 had received placebo and 56 had received eculizumab during REGAIN. Patients experienced rapid improvements in total scores and all four domain scores of both the myasthenia gravis activities of daily living profile and the quantitative myasthenia gravis scale with eculizumab treatment. These improvements were sustained through 130 weeks of the open-label extension. Interpretation: Eculizumab treatment elicits rapid and sustained improvements in muscle strength across ocular, bulbar, respiratory, and limb/gross motor muscle groups and in associated daily activities in patients with refractory anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.
publishDate 2020
dc.date.none.fl_str_mv 2020
dc.type.none.fl_str_mv info:eu-repo/semantics/article
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv https://hdl.handle.net/2445/196128
url https://hdl.handle.net/2445/196128
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv Reproducció del document publicat a: https://doi.org/10.1002/acn3.51121
Annals of Clinical and Translational Neurology, 2020, vol. 7, num. 8, p. 1327-1339
https://doi.org/10.1002/acn3.51121
dc.rights.none.fl_str_mv cc-by-nc-nd (c) Mantegazza, Renato et al., 2020
https://creativecommons.org/licenses/by-nc-nd/4.0/
info:eu-repo/semantics/openAccess
rights_invalid_str_mv cc-by-nc-nd (c) Mantegazza, Renato et al., 2020
https://creativecommons.org/licenses/by-nc-nd/4.0/
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv American Neurological Association
publisher.none.fl_str_mv American Neurological Association
dc.source.none.fl_str_mv Articles publicats en revistes (Ciències Clíniques)
reponame:Dipòsit Digital de la UB
instname:Universidad de Barcelona
instname_str Universidad de Barcelona
reponame_str Dipòsit Digital de la UB
collection Dipòsit Digital de la UB
repository.name.fl_str_mv
repository.mail.fl_str_mv
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score 15,300719

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